A Phase II, randomised, multi-centre placebo-controlled, double-blind study to investigate the safety of GS-248, and efficacy on Raynaud’s phenomenon (RP) and peripheral vascular blood flow in patients with systemic sclerosis (SSc)

Gesynta Pharma AB0 个研究点目标入组 80 人开始时间: 2020年9月30日最近更新: 2021年5月10日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Gesynta Pharma AB
入组人数: 80

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional clinical trial of medicinal product

入排标准

性别: All

入选标准

•1. Subjects must provide signed and dated written informed consent before the conduct of any study-specific procedures.
•2. Male and female subjects aged 18-75 years inclusive.
•3. SSc diagnosed according to European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) criteria (van den Hoogen F et al. 2013).
•Subjects with signs of other autoimmune diseases (e.g. Sjögren’s syndrome, myositis, rheumatoid arthritis) could be included if SSc is the dominating phenotype.
•4. Raynaud attacks typically =7 times per week during the last 4 weeks prior to screening despite background medication (only allowed vasodilatory therapy is calcium channel blockers or PDE-5 inhibitors).
•5. Women of childbearing potential (WOCBP) must be using a highly effective method of contraception to avoid pregnancy throughout the study and for 4 weeks after the last dose of IMP in such manner that the risk of pregnancy is minimised.*
•6. Women must not be pregnant or breastfeeding.
•7. Male subjects to agree to use condom in combination with use of contraceptive methods with a failure rate of <1% to prevent pregnancy and drug exposure of a partner and refrain from donating sperm from the first date of dosing until 3 months after last dosing of the IMP.
•8. Ability of subjects to participate fully in all aspects of this clinical trial.
•* A woman is considered of childbearing potential, i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
•*Methods that can achieve a failure rate of <1% per year when used consistently and correctly are considered as highly effective birth control methods. Such methods include combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, Intravaginal, and/or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, and/or implantable2); intrauterine device (IUD)2; intrauterine hormone-releasing system (IUS); bilateral tubal occlusion2; vasectomised partner; sexual abstinence.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 65
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 15

排除标准

•1. SSc disease duration of greater than 120 months from first non-Raynaud manifestation
•2. Current smokers or stopped smoking or used nicotine in any form <3 months prior to Visit 1.
•3. Dose-change or initiation of vasodilating substances (calcium blockers or PDE-5 inhibitors) within 4 weeks prior to Visit 1. Subjects are not allowed to use a combination of calcium blockers and PDE-5 inhibitors from 4 weeks prior to Visit 1 and throughout the study.
•4. Use of iloprost or other intravenous (iv) or per os (po) prostacyclin receptor agonist within 4 weeks prior to Visit 1.
•5. Ongoing treatment with immunosuppressive therapies (other than mycophenolate) including, but not restricted to; cyclophosphamide, azathioprine, methotrexate, or cyclosporine, or use of those medications within 4 weeks of trial entry prior to Visit 1.
•Note: Subjects could be included if they have been treated with a stable dose of mycophenolic acid during 4 weeks prior to study entry.
•6. Use of systemic corticosteroids within 4 weeks prior to Visit 1 and during the course of the study.
•7. Concurrent serious medical condition, with special attention to cardiovascular conditions, which in the opinion of the Investigator makes the subject not suitable for this study.
•8. Prolonged corrected QT interval by Fridericia (QTcF) defined as mean QTcF >450 msec at Visit 1, or at Visit 2 (prior randomisation).
•9. Creatinine clearance <50 mL/min (determined by Cockcroft-Gault equation).
•10. Active digital ulcer (DU) within 4 weeks prior to Visit 1.
•11. Have known allergies to any components of the GS-248 formulation.
•12. Clinically meaningful laboratory abnormalities at Screening (Visit 1), as determined and documented by the Investigator.
•13. Positive test results for HBsAg, HCVAb or HIV-1 and/or -2 antibodies at Screening (Visit 1).
•14. Subjects known or suspected of not being able to comply with this trial protocol (e.g. due to alcoholism, drug dependency or psychological disorder).
•15. Subject is mentally or legally incapacitated at the time of screening or has a history of clinically significant psychiatric disorders that would impact the subject’s ability to participate in the study according to the Investigator.
•16. Malignancy within the past 5 years except for in situ removal of basal cell carcinoma and cervical intraepithelial neoplasia grade I.
•17. Planned major surgery within the duration of the study.
•18. Blood donation (or corresponding blood loss) within 12 weeks prior to Visit 1
•19. Participation in another interventional clinical study involving IMP within 4 weeks or given an experimental drug within 5 half-lives, whichever longest, prior to Visit 1.
•Exclusion Criterion for Cold Challenge:
•At Visit 2: Finger temperature below 27°C after acclimatising at an ambient temperature of 23°C (±2°C) for a period of 20 minutes.
•Randomization Criteria:
•In addition to fulfilling all inclusion and exclusion criteria, subjects must fulfil the following criteria to be randomised:
•1. =7 RP attacks during the last week of the run-in period as captured in the eDiary, with no more than 2 days without RP attacks.
•2. Compliance with the eDiary during the 7 most recent days prior to baseline (Visit 2), excluding the visit day itself, defined as having submitted =5 days of eDiary records (out of a possible 7 days) for RCS and RP during that period.

研究者

发起方

Gesynta Pharma AB

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